iiinova — where your digital self keeps evolving

DMD WarrioR @dmdwarrior.com · 31/01/2026

SonoThera’s RIPPLE Technology Offers Hope for Full-Length Human Dystrophin 👉 Read More: DMDWarrioR.com

SonoThera’s RIPPLE Technology Offers Hope for Full-Length Human Dystrophin

SonoThera, a biotechnology company dedicated to treat the root causes of human diseases by developing the next generation of genetic medicines, announced on January 8, 2026, that its proprietary RIPPLE™ technology had detected strong and persistent full-length human dystrophin protein expression in the skeletal muscles of non-human primates, reaching up to 290% of normal levels.

To learn more about SonoThera’s RIPPLE technology firsthand, we contacted Elizabeth Harness from the Corporate Communications department and shared our questions on behalf of our followers. Elizabeth thoughtfully and comprehensively provided details about Ultrasound-Mediated Delivery (UMD) technology, which is also planned for use in Duchenne muscular dystrophy.

DMD WarrioR @dmdwarrior.com · 12/12/2025

Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing #saRNA Therapeutic for #Duchenne Muscular Dystrophy 👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 11/12/2025

Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy 👉 Read More: DMDWarrioR.com

Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy
Atossa Therapeutics announced that the U.S. Food and Drug Administration (“FDA”) has granted Rare Pediatric Disease (“RPD”) designation to (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy (“DMD”).
👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 09/12/2025

Satellos reported that the FDA and other global regulators have cleared its IND for SAT-3247, enabling a three-month Phase 2, placebo-controlled trial in 51 ambulatory children with Duchenne muscular dystrophy. #satellos #dmd #duchenne 👉 Read More: DMDWarrioR.com

Satellos Receives FDA and International Clearance to Commence Pediatric Phase 2 Testing of SAT-3247 for Duchenne Muscular Dystrophy
Satellos reported that the FDA and other global regulators have cleared its IND for SAT-3247, enabling a three-month Phase 2, placebo-controlled trial in 51 ambulatory children with Duchenne muscular dystrophy. #satellos #dmd #duchenne
👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 26/11/2025

Researchers Buel D. Rodgers and Christopher W. Ward published a groundbreaking study in Gene Therapy that unveiled a codon-optimized human Smad7 gene therapy aimed at treating Duchenne muscular dystrophy (DMD). 👉 Read More: DMDWarrioR.com

Researchers Develop Optimized Smad7 Gene Therapy to Target Duchenne Muscular Dystrophy

Researchers Buel D. Rodgers and Christopher W. Ward published a groundbreaking study in Gene Therapy that unveiled a codon-optimized human Smad7 gene therapy aimed at treating Duchenne muscular dystrophy (DMD).

👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 25/11/2025

ELEVIDYS Gene Therapy for Non-Ambulant Children with DMD: FDA OKs ENDEAVOR Cohort 8 Immunosuppression Study 👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 18/11/2025

Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature 👉 Read More: DMDWarrioR.com

Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature
Satellos Therapeutics announced that researchers at the Ottawa Hospital Research Institute have published new findings in Nature Communications validating the company’s innovative approach to treating the root cause of Duchenne muscular dystrophy and related diseases.
👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 17/11/2025

DMD ANKA mRNA therapy, currently under development for Duchenne muscular dystrophy in Turkiye, was announced by Associate Professor Dr. Ali Taghizadeh. 👉 Read More: DMDWarrioR.com

Ali Taghizadeh Announces DMD ANKA mRNA Therapy, Currently in Clinical Trial in Turkiye
DMD ANKA mRNA therapy, currently under development for Duchenne muscular dystrophy in Turkiye, was announced by Associate Professor Dr. Ali Taghizadeh.

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DMD WarrioR @dmdwarrior.com · 30/10/2025

REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program #dmd #duchenne #regenxbio #genetherapy #clinicaltrials #rgx202 👉 Read More: DMDWarrioR.com

REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program
REGENXBIO announced it has completed enrollment of 30 participants in the pivotal portion of its AFFINITY DUCHENNE trial for RGX-202, an investigational gene therapy for Duchenne muscular dystrophy.

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DMD WarrioR @dmdwarrior.com · 30/10/2025

Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy #dmd #duchenne #imlifidase #genetherapy #clinicaltrials 👉 Read More: DMDWarrioR.com

Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy
Two gene therapy trials, one sponsored by Sarepta and the other sponsored by Genethon, support the ability of imlifidase to substantially reduce anti-AAV antibodies to allow administration of gene therapy.

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DMD WarrioR @dmdwarrior.com · 22/10/2025

Why Elevidys Was Not Approved by the European Medicines Agency (EMA)? You can access the comprehensive report on the reasons for the Elevidys refusal published by EMA from our web page link. #dmd #duchenne #elevidys #eu 👉 Read More: DMDWarrioR.com

If you're searching for answers to 'why Elevidys was not approved by the European Medicines Agency (EMA)', you're not alone. In this article, we explore the key reasons behind the EMA’s decision and what it means for patients, families, and the future of gene therapy in the EU.

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DMD WarrioR @dmdwarrior.com · 14/10/2025

Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping #dmd #duchenne #exon44 #brogidirsen 👉 Read More: DMDWarrioR.com

Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping

Nippon Shinyaku announced efficacy and safety data from a 3.5-year application based on an open-label extension study that also included the initiation of a clinical trial for brogidirsen (NS-089/NCNP-02).

#dmd #duchenne #exon44 #brogidirsen

👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 13/10/2025

Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026 #dmd #duchenne #delzota #exon44 #exon44skipping #clinicaltrials #aviditybiosciences 👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 13/10/2025

Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production? #pemf #dmd #duchenne 👉 Read More: DMDWarrioR.com

Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production?
In countries like Uzbekistan, information is being shared about the potential treatment of Duchenne Muscular Dystrophy with PEMF therapy. Pulsed Electromagnetic Field (PEMF) therapy is not a cure for Duchenne Muscular Dystrophy. Be careful not to let scammers take your money.

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DMD WarrioR @dmdwarrior.com · 30/09/2025

Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy #dmd #duchenne

Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy

Tevard Biosciences announced the presentation of new preclinical data showing potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM-TTNtv).

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DMD WarrioR @dmdwarrior.com · 28/09/2025

Cures of #Duchenne (Detailed List of All Clinical Trials for #DMD) List of all researches such as #gene therapy, exon skipping, reducing inflammation and improving #muscle growth & protection cures for duchenne #muscular dystrophy. dmdwarrior.com/dmd-treatmen...

Cures of Duchenne (Detailed List of All Clinical Trials)

List of all researches such as gene therapy, exon skipping, reducing inflammation and improving muscle growth & protection cures for duchenne muscular dystrophy.

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DMD WarrioR @dmdwarrior.com · 10/09/2025

Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial #dmd #duchenne #delzota #exon44 #exon44skipping #clinicaltrials #aviditybiosciences 👉 Read More: DMDWarrioR.com

Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial

With del-zota, normal dystrophin increased by 25% and creatine kinase decreased by 80%. Among exon 44 skipping therapies, the highest values currently received have caused excitement.

Unprecedented rapid reduction in creatine kinase (CK) to near normal levels maintained over 16 months of follow-up and 25% increase of normal in dystrophin production, reflecting sustained muscle fiber protection.

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DMD WarrioR @dmdwarrior.com · 09/09/2025

Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy #dmd #duchenne #BBMD101 #BeliefBioMed #clinicaltrials 👉 Read More: DMDWarrioR.com

Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy
Belief BioMed, an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that the registration clinical trial (CTR20252461) of its independently developed and produced BBM-D101 injection for Duchenne Muscular Dystrophy (DMD) has successfully completed the first patient dosing in Beijing, China.

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DMD WarrioR @dmdwarrior.com · 09/09/2025

Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males #dmd #duchenne #sgt003 #genetherapy #clinicaltrials 👉 Read More: DMDWarrioR.com

Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males

The phase 3 study (NCT07160634) of the SGT-003 gene therapy developed by Solid Biosciences is expected to begin in October 2025 and be completed in January 2029. The SGT-003 Phase 3 study will include 80 ambulant males, moreover the study locations have not yet been shared.

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DMD WarrioR @dmdwarrior.com · 08/09/2025

Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting #dmd #duchenne #ker065 👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 07/09/2025

GNR-097: Duchenne Gene Therapy Phase 1/2 Study Started by Generium Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD). 👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 06/09/2025

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping

🎈 SEPTEMBER 7, 2025 🎈

Support World Duchenne Awareness Day 2025 with DMDWarrior

Theme of 2025: Make Cures Affordable for Duchenne

We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior.

Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure.

🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact:

#WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD
#UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping

💪 Together We Are Stronger

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DMD WarrioR @dmdwarrior.com · 06/09/2025

How Does My Child Participate in Clinical Trials for Duchenne? Learn how your child can join Duchenne muscular dystrophy (DMD) clinical trials and discover trusted resources to find active studies near you. Learn More 👇 dmdwarrior.com/how-to-parti...

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DMD WarrioR @dmdwarrior.com · 30/08/2025

Support World Duchenne Awareness Day 2025 with DMDWarrior

World Duchenne Awareness Day will be observed globally on September 7, 2025, with the theme “Make Cures Affordable for Duchenne.” This important day raises awareness about Duchenne Muscular Dystrophy (DMD), a rare genetic condition that causes progressive muscle weakness and affects thousands of individuals and families worldwide. Each year, the campaign calls for more attention, research, and action toward better treatments and, ultimately, a cure.

Theme of 2025: Make Cures Affordable for Duchenne

This year’s theme reflects the urgent and universal wish of the Duchenne community: access to effective and affordable cures for Duchenne Muscular Dystrophy (DMD). Families, patients, and advocacy groups are united in calling for equitable access to treatments, accelerated research, and global collaboration.

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DMD WarrioR @dmdwarrior.com · 25/08/2025

ITF Therapeutics Announces Publication of Positive Long-Term Data Reinforcing Duvyzat (Givinostat) Efficacy and Safety as a Treatment for Duchenne Muscular Dystrophy #givinostat #duvyzat 👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 19/08/2025

Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026 #agamree #vamorolone #uae #bahrain #oman #saudi_arabia #kuwait 👉 Read More: DMDWarrioR.com

Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026

Santhera Pharmaceuticals announces the signing of an exclusive agreement with Uniphar to manage the distribution of AGAMREE (vamorolone) in the United Arab Emirates, the Kingdom of Saudi Arabia, Kuwait, Oman and Bahrain, for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older.

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DMD WarrioR @dmdwarrior.com · 29/07/2025

FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’ #dmd #sarepta #roche #elevidys #fda #duchenne 👉 Read More: DMDWarrioR.com

FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’

In a statement Monday, it said the FDA's swift action eliminated a problem with Sarepta, allowing Elevidys to return to the market without another safety study as had been feared.

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DMD WarrioR @dmdwarrior.com · 26/07/2025

FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys #dmd #sarepta #roche #elevidys #fda #duchenne 👉 Read More: DMDWarrioR.com

FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys

The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and voluntary suspension of product distribution as it investigates the safety concerns.

The U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy.

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DMD WarrioR @dmdwarrior.com · 25/07/2025

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys #dmd #sarepta #roche #elevidys #europe #ema 👉 Read More: DMDWarrioR.com

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys

A European Medicines Agency committee ruled that the therapy, called Elevidys, failed to show in studies that it improved patients’ movement abilities.

Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).

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DMD WarrioR @dmdwarrior.com · 23/07/2025

Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States #dmd #sarepta #roche #elevidys 👉 Read More: DMDWarrioR.com

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DMD WarrioR @dmdwarrior.com · 15/06/2025

Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients. #dmd #sarepta #roche #elevidys 👉 Read More: DMDWarrioR.com

Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients.

Sarepta reports that Shipments of ELEVIDYS for infusions in non-ambulatory patients in commercial setting are suspended until enhanced regimen is approved and in place.

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DMD WarrioR @dmdwarrior.com · 28/05/2025

Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy #dmd #duchenne 👉 Read More: DMDWarrior.com

Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy

Entrada Therapeutics Receives Authorization in the European Union to Initiate ELEVATE-45-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-45 in Patients Living with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping.

Entrada Therapeutics, today announced it has received authorization from the Health Authorities and Ethics Committees of multiple countries under the European Union Clinical Trial Regulation (EU-CTR) to initiate ELEVATE-45-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-45 in patients living with Duchenne muscular dystrophy (DMD) who are amenable to exon 45 skipping.

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DMD WarrioR @dmdwarrior.com · 05/05/2025

Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy 👉 DMDWarrioR.com #dmd #duchenne #deramiocel #cardiomiopathy

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DMD WarrioR @dmdwarrior.com · 30/04/2025

Tevard Biosciences to Present Data Demonstrating tRNA-based Therapy Rescued Full-Length Dystrophin and Motor Function in Duchenne Muscular Dystrophy Model 👉 DMDWarrioR.com #dmd #duchenne #tRNA #Dystrophin

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DMD WarrioR @dmdwarrior.com · 25/04/2025

EMA Recommends EU Approval for Duvyzat (givinostat) to Treat Duchenne Muscular Dystrophy #dmd #duchenne #ema #duvyzat #givinostat 👉 DMDWarrioR.com

EMA Recommends EU Approval for Duvyzat (givinostat) to Treat Duchenne Muscular Dystrophy
EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Duvyzat (givinostat) as a treatment for Duchenne muscular dystrophy (DMD) in patients from the age of six who are able to walk.
The European Medicines Agency (EMA) has suggested that Duvyzat (givinostat) be given a conditional marketing authorization in the EU to treat Duchenne muscular dystrophy (DMD) in individuals who can walk starting at age six. Duvyzat is a suspension that is given orally in conjunction with corticosteroid therapy.

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DMD WarrioR @dmdwarrior.com · 24/04/2025

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy #dmd #duchenne #dyne #dyne-251 #exon51 #exon51skipping #exonskipping #ema #orphandrug

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy

Dyne Therapeutics, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD).

Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated as an orphan drug by the European Commission (EC) for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being tested in DMD patients who are susceptible to exon 51 skipping in the Phase 1/2 DELIVER global clinical study.

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DMD WarrioR @dmdwarrior.com · 23/04/2025

Russia-Based Circle of Kindness Foundation Announces 18% Price Cut for Elevidys Gene Therapy #dmd #elevidys #russia #duchenne #dmdwarrior 👉 Read More: DMDWarrioR.com

Russia-Based Circle of Kindness Foundation Announces 18% Price Cut for Elevidys Gene Therapy

As a result of the negotiations, the price of one of the most expensive drugs in the world was reduced by 18 percent, or 400 thousand euros, to 2.2 million euros," the press service of the Circle of Kindness Charitable State Foundation said.

According to the Foundation’s press office, “as a result of the negotiations, the price of one of the most expensive drugs (Elevidys) in the world was reduced by 18%, that is by €400,000, to €2.2 million.”

Take from the Rich, Give to the Poor
Russia has set up a foundation called “Circle of Kindness,” which is financed by 2% of the individual income tax collected by high-income earners, despite the fact that rare (orphan) diseases pose a substantial financial burden on health systems and that access to these costly therapies is restricted. Since its founding in 2021, this sustainable mechanism has enabled the provision of medications for more than 24,000 children with rare diseases in 84 regions of the Russian Federation, totaling more than $2,4 billion USD. Resource allocation decisions are made using the process of health technology assessment.

2% of the high-income earners’ individual income taxes go toward funding the “Circle of Kindness” Foundation. Stated differently, each Russian citizen who earns more than 5 million rubles a year donates 2% of their taxes to the foundation. In addition to ensuring that children with rare and life-threatening illnesses have access to orphan medications, this dependable and sustainable financing method also removes the need for ongoing community fundraising initiatives.

The “Circle of Kindness” has helped over 24,000 children in 84 locations since it was founded, spending 226,000,000 rubles ($2.4 billion USD equivalent) on this much-needed treatment.

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DMD WarrioR @dmdwarrior.com · 06/04/2025

mRNA Studies in DMD Treatment: Future Therapeutic Candidates Duchenne Muscular Dystrophy (DMD) mRNA Studies May Provide Full-Length and Normal Dystrophin Production. #dmd #mrna #duchenne #dmdwarrior 👉 Read More: DMDWarrioR.com

Duchenne Muscular Dystrophy (DMD) mRNA Studies May Provide Full-Length and Normal Dystrophin Production.
Given that FDA-approved exon skipping therapies and gene therapy for Duchenne Muscular Dystrophy, which are known to not produce sufficient levels of dystrophin and thus do not completely eliminate Duchenne Muscular Dystrophy (DMD). Over the years, treatment options for DMD have been limited, but a promising area of research has emerged with the use of mRNA technology. These studies may enable production of full-length and normal dystrophin for DMD disease.

Although exon skipping cures and gene therapy have recently been approved by the US Food and Drug Administration (FDA), none of these are thought to completely cure DMD, and there remains an urgent need to create methods that restore myofiber integrity, reverse the loss of muscle regenerative capacity, and address mitochondrial dysfunction, none of which are offered by current treatments.

About 1 in 5,000 male newborns are affected by DMD, one of the most severe muscle degeneration illnesses. It is brought on by mutations in the X chromosome-related dystrophin gene. Young males with DMD experience gradual muscular degeneration, inflammation, fibrosis, and eventually pass away from heart and respiratory failure.

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DMD WarrioR @dmdwarrior.com · 01/04/2025

Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe Why Were Elevidys Clinical Trials Halted? 👉 DMDwarrior.com

Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe

Following the death of a 16-year-old child receiving an Elevidys infusion, 3 ongoing clinical trials in Europe (Study 104, Study 302 and Study 303) were temporarily halted.

Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta and marketed by Roche, have been temporarily halted by the European Medicines Agency (EMA).

Why Were Elevidys Clinical Trials Halted?

After the death of a 16-year-old boy who received an Elevidys infusion on March 18, 2025, the perspective on the gene therapy marketed at a commercial price of $3 million suddenly changed.

👉 DMDwarrior.com

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DMD WarrioR @dmdwarrior.com · 06/03/2025

GenAssist Announces FDA Approval of New Drug Application for GEN6050X Exon 50 Skipping Therapy 👉 Read More: dmdwarrior.com #dmd #duchenne #GenAssist #GEN6050X #exon50 #exon50skipping

GenAssist Announces FDA Approval of New Drug Application for GEN6050X Exon 50 Skipping Therapy

On March 06, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, is thrilled to announce that it has received clearance from the U.S Food and Drug Administration(FDA) for its Investigational New Drug (IND) application for GEN6050X, a first-in-class base editing drug for Duchenne Muscular Dystrophy (DMD). GenAssist is planning to conduct clinical study globally for GEN6050X.

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DMD WarrioR @dmdwarrior.com · 04/03/2025

Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy 👉 Read More: dmdwarrior.com #dmd #duchenne #deramiocel #capricor

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DMD WarrioR @dmdwarrior.com · 24/02/2025

Entrada Therapeutics Receives FDA Approval to Begin Clinical Trial of ENTR-601-44 Exon Skipping Treatment 👉 Read More: dmdwarrior.com #dmd #duchenne #exon44 #exon44skipping #exonskipping

Entrada Therapeutics Receives FDA Approval to Begin Clinical Trial of ENTR-601-44 Exon Skipping Treatment

Entrada Therapeutics, announced that the United States Food and Drug Administration (FDA) has lifted the clinical hold on ENTR-601-44 and provided authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose (MAD) clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in adult patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping.

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DMD WarrioR @dmdwarrior.com · 18/02/2025

Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 👉 Read More: dmdwarrior.com #dmd #duchenne #solidbio #sgt-003 #genetherapy

Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003

Solid Biosciences, a clinical-stage biotechnology company, shared positive initial data from its Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy for Duchenne muscular dystrophy.

Clinical Data of Duchenne Gene Therapy Candidate SGT-003

📌Average microdystrophin expression of 110% (N=3) and significant improvements in multiple additional muscle health biomarkers observed support the potential of SGT-003 as a next-generation, best-in-class Duchenne muscular dystrophy gene therapy candidate.

📌Encouraging early signals of potential cardiac benefit observed.

📌SGT-003 has been well-tolerated in the 6 participants dosed as of February 11, 2025, with no serious adverse events observed.

📌Participant enrollment continues, with the 7th participant dosed on February 17, 2025; Company expects to dose approximately 20 total participants by Q4 2025.

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DMD WarrioR @dmdwarrior.com · 16/02/2025

We Call on Health Ministries and Pharmaceutical Manufacturers to Take Action on Duchenne Treatments Who is Guilty? 👉 Read More: dmdwarrior.com #dmd #dmdwarrior #duchenne @fda.gov @ema.europa.eu @ec.europa.eu @who.int @anvisaoficial.bsky.social

We Call on Health Ministries and Pharmaceutical Manufacturers to Take Action on Duchenne Treatments

Doesn't it upset you that children in your country are leaving this world because they can't access the necessary treatments? You are to blame. We call on all health ministries around the world and manufacturers of DMD treatments approved by the FDA and EMA to take action.

It’s heart-wrenching to think that children in today’s world, in countries across the globe, are losing their lives far too early simply because they cannot access the medical treatments they so desperately need. This is the tragic reality for many children suffering from Duchenne Muscular Dystrophy (DMD), a devastating and rare genetic condition that leads to progressive muscle weakness and, ultimately, early death. The fact that life-saving treatments exist, yet these children are still dying due to a lack of access, is something that we cannot ignore. And the harsh truth is, we are all responsible for this crisis.

Who is Guilty?

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DMD WarrioR @dmdwarrior.com · 16/02/2025

Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments 👉 Read More: dmdwarrior.com #dmd #dmdwarrior #duchenne

Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments

While children with DMD are counting down the days until they regain their health, pharmaceutical companies are calculating their year-end revenues. This is what they call the Duchenne Muscular Dystrophy Market. If you have money, you can shop at this market. If you don't have money, your children are left alone to their fate.

While DMD families struggle to restore their children to health, pharmaceutical companies whose primary mission is to heal children are focused on year-end profits. While research companies analyze the Duchenne Muscular Dystrophy market, pharmaceutical companies calculate their income. No one thinks about the children who will not have access to medicine.

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DMD WarrioR @dmdwarrior.com · 14/02/2025

Santhera Announces Agreement with German GKV-SV on Reimbursement Amount for AGAMREE (vamorolone) for the Treatment of Duchenne Muscular Dystrophy in Germany 👉 Read More: dmdwarrior.com

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DMD WarrioR @dmdwarrior.com · 10/02/2025

The Challenge of Getting DMD Gene Therapy: Is Geography Destiny? #duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness 👉 Read More: dmdwarrior.com

The Challenge of Getting DMD Gene Therapy: Is Geography Destiny?

Over the last decade, significant advancements have been made in the development of gene therapies for DMD, offering hope for a life-changing treatment. However, despite the promise these therapies hold, a major obstacle stands in the way of many patients: the prohibitively high cost of these treatments. This article explores the reasons behind the exorbitant price of Duchenne gene therapies and the devastating consequences for patients, families, and the healthcare system as a whole.

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DMD WarrioR @dmdwarrior.com · 10/02/2025

Satellos Announces Completed Enrollment of Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers #duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness #satellos 👉 Read More: dmdwarrior.com

Satellos Announces Completed Enrollment of Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers

SAT-3247 Announcement Summary

📌Company remains on track to report Phase 1a data from both the Single- and Multiple-Ascending Dose (SAD and MAD) cohorts in the healthy volunteer portion of the study in 1Q 2025 at an upcoming major medical meeting

📌The Phase 1b portion of the trial, in DMD patients, is underway with the intention of enrolling up to 10 adult volunteers with genetically confirmed DMD during 1Q 2025

📌Phase 2 IND filing on track to be submitted by end of 1Q 2025

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DMD WarrioR @dmdwarrior.com · 04/02/2025

Cumberland Pharmaceuticals Announces Fantastic Results from Phase 2 FIGHT Duchenne Trial in DMD Heart Disease What is Ifetroban? 👉 Read More: dmdwarrior.com

Cumberland Pharmaceuticals Announces Fantastic Results from Phase 2 FIGHT Duchenne Trial in DMD Heart Disease

Cumberland Pharmaceuticals announced results from its Phase 2 FIGHT DMD study (Ifetroban), which continues to develop the drug for Duchenne Muscular Dystrophy Heart Disease.

What is Ifetroban?

Ifetroban is a once-daily oral medication that works by blocking the thromboxane receptor, which plays a key role in inflammation and fibrosis. The drug has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, highlighting its potential significance in treating this devastating condition. If approved, ifetroban would be the first therapy specifically indicated for DMD-related heart disease.

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DMD WarrioR @dmdwarrior.com · 03/02/2025

Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping) 👉 Read More: dmdwarrior.com #duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness #uk #england #exon44skipping #entrada

Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping)

Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping Treatment), a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-44 in Duchenne Muscular Dystrophy Patients.

ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44, was authorized by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee for the Clinical Trial of an Investigational Medicinal Product. The study aims to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 44 skipping.

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